Gm2 Gangliosidosis
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis [Phase 3]
A Phase 3 trial is evaluating oral Nizubaglustat (AZ-3102) for late-infantile and juvenile GM1 gangliosidosis and GM2 gangliosidosis, and th…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis [Phase 3]
This clinical trial update notes that oral Nizubaglustat (AZ-3102) is being studied in a Phase 3 trial for late-infantile and juvenile Niema…
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
A Phase 2 clinical trial is evaluating the safety and efficacy of Nizubaglustat (AZ-3102) in patients with GM2 gangliosidosis and Niemann-Pi…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: A Natural History Study of the Gangliosidoses
This clinical trial update describes an ongoing natural history study of gangliosidoses. It includes Tay-Sachs disease, Sandhoff disease, GM…
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: A Natural History of Late Onset Tay-Sachs Disease
A completed natural history study of GM2 gangliosidosis, including late-onset Tay-Sachs disease, sponsored by Massachusetts General Hospital…
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CLINICAL_TRIALS research · 2025-03-20Clinical Trial Update: Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
Phase 2 study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of oral AZ-3102 in GM2 gangliosidosis and Niemann-Pick …